Outcomes and Healthcare Resource Utilisation in Adults With von Willebrand Disease Receiving On‐Demand Recombinant von Willebrand Factor in the United Kingdom

Le résumé fourni par la source

OBJECTIVES: We describe treatment outcomes and healthcare resource utilisation (HCRU) in adults with von Willebrand disease (VWD) treated on demand with recombinant von Willebrand factor (rVWF) in the United Kingdom. METHODS: Retrospective chart review of adults (≥ 18 years) with congenital VWD receiving first-time rVWF for the on-demand treatment of spontaneous/traumatic bleeds, or the prevention/treatment of surgical bleeds, between 1 October 2020 and 30 June 2022 at seven hospitals. Treatment, outcome and VWD-related HCRU data associated with the on-demand treatment of all recorded bleeds were collected at first (index) treatment, for 12 months pre-index and 3-12 months post-index. RESULTS: Twelve patients (91.7% female and White/Caucasian, mean age 34.2 years) were treated on demand with rVWF at index: bleed resolution was achieved for all bleeds with limited requirement for additional treatments, no treatment switches, and no complications reported. Physician-rated treatment satisfaction was 'excellent' (82.6%) or 'good' (17.4%) for all recorded on-demand rVWF-treated bleeds (n = 23). Patients receiving any on-demand treatment required an inpatient admission and ≥ 1 outpatient visit in 33.7% and 66.7% of cases, respectively. CONCLUSIONS: Results support the effectiveness and safety profile of on-demand rVWF treatment for spontaneous/traumatic bleeds in adults with VWD, adding to the growing body of evidence for rVWF.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.